The long Journey from Molecule to Medicine

A point of view by Tine Laethem, AVP Translational Pharmacology Europe Inc, Brecht Vanneste, AVP Managing Director MSD Belgium & Luxembourg and Erik Antonissen, Clinical Operations Director

Medicines don’t grow on trees

Medicines don’t grow on trees. It takes about thirteen years of research and studies, and between two and three billion euros to get from 10.000 molecules to one medicine that reaches the patient. That long journey is necessary to make sure that the new medicine you will receive is safe and efficient.

From 10.000 to 10: Preclinical trials

The journey of finding a new treatment starts with deciding what disease we will focus on. We need to screen between 10.000 and 30.000 molecules to find the ones that interfere with the cells causing the disease – and are also safe for humans. We put those molecules through a series of tests, and at the end of this stage, we’ll be left with about ten preclinical candidates.

Phase I: Clinical trials on healthy volunteers

In phase one of clinical testing, we’ll give the candidate-medicine to a small group of healthy volunteers. We want to verify if the medicine is safe, and whether there is any effect on the body.

Phase II and phase III: Clinical trials on patients

When the phase I-results are favorable, we move on to phase II: we test the medicine on a small group of patients, to see if there is any effect on the disease. If the candidate-medicine is effective, we scale up the clinical study program. This is phase III: a bigger group of patients over a longer period. This is important to check the long-term effects: what happens when you give the candidate-medicine to 1.000, 10.000 or even 30.000 patients over the course of two to five years?

In these clinical trials, we compare our candidate to either a placebo or the standard of care: the current treatment for patients with this disease. The goal is to identify if our medicine has an advantage on what is on the market now: is it more effective? Are there fewer side effects? Does it allow for easier administration? In other words, is there any innovation for the patient? This is the point where nine out of ten candidate medicines fail: not only because of their efficacity or safety, but potentially also because they’re not significantly better than what is available on the market.

Getting approved

Once we have completed a successful phase III trial, we can submit a request for approval to the European Medicines Agency (EMA). This procedure takes about a year, and they will decide if our medicine is granted access to the market. Once we secure our European approval, we can submit a request in every member country to get an approval on price and reimbursement. In Belgium, it’s the National Institute for Health and Disability Insurance (RIZIV/INAMI) that will decide.

«It took almost 25 years between the discovery of the hepatitis C virus and the launch of safe, tolerable, and simple treatments that deliver over 95% cure rates¹»

Belgium has a drive for innovation

Belgium is a world leader in clinical trials: this is thanks to our favorable regulatory environment – which ensures fast and tight deadlines for the approval and roll-out of clinical trials – and the high number of extremely qualified academics, especially in the field of oncology. Our country has a drive for innovation and that comes with a lot of benefits: our patients get early access to new and innovative medication, and the industry is responsible for 37.000 jobs. At this moment, MSD Belgium is running more than 50 clinical trials. Over 70% of them are in the field of oncology, and 95% of those are in immuno-oncology. Our new types of medicines called immuno-boosters give the immune system a boost to recognize and attack cancer cells in the body. These innovative new medicines are already approved to treat several types of cancer, and our MSD clinical research program continues for over 40 different types of cancer in over 1.000 studies. This is a race that one day will result in a cure for cancer.

The cost of medicines

As a patient, when you take an innovative, life-prolonging medicine, it’s good to be aware of what has happened to get that medicine to you: the molecules that we tested but didn’t work, the research and trials that we conducted for ten to fifteen years and the cost of the whole process to get to that one medicine that is saving your life today. To be able to do it all over again for other possible cures, we need to invest heavily in new research. There’s still a lot of areas where there’s no satisfying treatment today: in Alzheimer’s disease or neuro-degenerative diseases to name a few. We cannot back away from research that has a very small chance of success, because our patients need us to find a treatment. At MSD, we ‘invent for life’!

1Adapted from Burstow et al., “Hepatitis C treatment: where are we now?”, International journal of general medicine, 10, 39–52,2017

BE-NON-00360. Date of last revision: 02/2020

Tine Laethem

AVP Translational Pharmacology Europe Inc

Brecht Vanneste

AVP Managing Director MSD Belgium & Luxembourg

Erik Antonissen

Clinical Operations Director